“Very Promising” Treatment for Huntington Disease

Researchers at the University of Alberta (U of A) have discovered a promising new therapy for Huntington disease that restores lost motor skills and may delay or stop the progression of the disease based on lab model tests, says the lead researcher.

Because the new therapy uses a molecule already being used in clinical trials for other diseases, it could be used in a clinical trial for Huntington disease within the next one to two years.

"We did not expect to see such dramatic changes after administering this therapy," said Simonetta Sipione, the Principal Investigator. "We expected to see improvement, but not complete restoration of motor skills. When we saw this, we were jumping with excitement in the lab. This is very promising and should give hope to those with Huntington disease. I think it's a treatment that deserves to go to clinical trials because it could have huge potential."

Those with this inherited brain disorder, where a mutant protein triggers brain cell death causing loss of motor and cognitive skills and eventually death, have slightly lower levels of a brain molecule known as GM1. When U of A medical researchers restored GM1 to normal levels in lab models with the disease, motor skills in the lab models returned to normal within days, said Sipione.

During the research stage, lab models at the U of A were given the GM1 molecule therapy for four weeks. During the first two weeks after the treatment finished, the lab models still had normal motor function. But after that, motor function started to decline and return to pre-treatment levels by the end of the fourth week. So a potential treatment with this molecule would involve repeated treatments over the long-term, says Sipione.

Sipione and her team are continuing their research to see if restored levels of the GM1 molecule can also reverse cognitive damage in lab models with Huntington disease. They hope to publish the results from these tests within one year. It seems the GM1 therapy improves the way neurons work and makes the mutant huntingtin protein less toxic.

"Because of the way it works, we think it will work on cognitive symptoms of the disease too," says Sipione.

REHACARE.de; Source: University of Alberta